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AL-S Pharma Reports Positive Phase 2 Results for AP-101 in ALS Treatment

Monoclonal AntibodyOrphan Drug
  • AL-S Pharma announced positive topline results from its Phase 2 study of AP-101, a first-in-class monoclonal antibody targeting misfolded SOD1 protein in amyotrophic lateral sclerosis patients.
  • The multicenter, randomized, double-blind, placebo-controlled study met its primary safety and tolerability endpoint in both sporadic ALS (N=52) and mutant SOD1-ALS (N=21) patients over 12 months of treatment.
  • Clinically meaningful changes in exploratory outcome measures related to survival and non-invasive ventilation were observed, along with stabilization of clinical disease-staging and neurofilament biomarkers.
  • This represents the first Phase 2 study to assess a SOD1-targeted therapeutic in both sporadic ALS and genetically determined SOD1-ALS patients, supporting the hypothesis that misfolded SOD1 protein plays a broader role in ALS pathogenesis.

A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT05039099CompletedPhase 2
AL-S Pharma
Posted 9/2/2021

Avadel Pharmaceuticals Acquires Exclusive Global Rights to Valiloxybate for Sleep Disorders

Orphan Drug
  • Avadel Pharmaceuticals has secured exclusive global licensing rights to valiloxybate from XWPharma for $20 million upfront, with potential milestone payments exceeding $205 million.
  • Valiloxybate represents the first salt-free, artificial sweetener-free, once-at-bedtime oxybate therapy designed for treating narcolepsy and idiopathic hypersomnia.
  • The company plans to initiate pharmacokinetic studies in Q4 2025, followed by pivotal trials in H2 2026, leveraging a streamlined bioequivalence pathway.
  • This acquisition strengthens Avadel's position in the expanding sleep medicine market, valued at $5.3 billion in 2024 and projected to reach $8.6 billion by 2033.

Biohaven Prepares for Potential First-in-Class Spinocerebellar Ataxia Drug Launch as FDA Decision Looms

Rare DiseaseDrug ApprovalOrphan Drug
  • Biohaven's Vyglxia could become the first approved treatment for spinocerebellar ataxia, a rare genetic brain disorder affecting movement and coordination, with FDA decision expected between October and December.
  • The company has established comprehensive launch infrastructure including patient hubs, insurance specialists, and sales teams, with CEO Vlad Coric stating they are "ready to ship on Day 1" following potential approval.
  • Approximately 400 patients are already receiving Vyglxia through expanded access programs, and the company estimates 6,000-7,000 of the 15,000 U.S. patients will be reachable at launch through specialized treatment centers.
  • Unlike previous competitive launches, Vyglxia faces no direct competition in the spinocerebellar ataxia market, positioning Biohaven for a streamlined commercial strategy with a sales team less than 10% the size used for their previous migraine drug.

Savara Reports 50% Increase in Autoimmune PAP Patient Population, Plans December 2025 FDA Resubmission for MOLBREEVI

Rare DiseaseOrphan Drug
  • Savara's updated health claims analysis identifies approximately 5,500 autoimmune PAP patients in the U.S., representing a 50% increase from the company's previous 2023 estimate of 3,600 patients.
  • The revised analysis used a more comprehensive dataset with over three times the number of records compared to the previous study, suggesting improved awareness and diagnosis rates for this rare lung disease.
  • The company plans to resubmit its Biologics License Application for MOLBREEVI to the FDA in December 2025 with priority review request, following publication of positive Phase 3 IMPALA-2 trial results in the New England Journal of Medicine.
  • With no approved medicines currently available in the U.S. for autoimmune PAP, the increased patient population estimate reinforces the significant unmet medical need for this debilitating condition.

Lantern Pharma Receives FDA Guidance for Pediatric CNS Cancer Trial Targeting ATRT

Orphan DrugAIRare Disease
  • Lantern Pharma completed a Type C meeting with the FDA, receiving critical guidance on the regulatory pathway and trial design for a planned pediatric CNS cancer trial targeting Atypical Teratoid Rhabdoid Tumor (ATRT).
  • The FDA confirmed the potential incorporation of spironolactone as a combination agent with LP-184/STAR-001 to assess synergistic effects in enhancing efficacy against CNS cancers.
  • The company's ATRT program has received both Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA, highlighting the urgent unmet medical need.
  • Lantern plans to submit an IND application amendment based on FDA guidance, with trial initiation targeted for Q1 2026.

Nanoscope's MCO-010 Receives FDA RMAT and Five EMA Orphan Designations for Retinal Dystrophies

Orphan Drug
  • The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to MCO-010 for Stargardt disease, building on prior Orphan Drug and Fast Track designations for both retinitis pigmentosa and Stargardt disease.
  • The European Medicines Agency awarded five Orphan designations to MCO-010 covering non-syndromic and syndromic rod-dominant and cone-dominant dystrophies, as well as macular dystrophies.
  • MCO-010 is a one-time intravitreal optogenetic therapy that activates bipolar retinal cells to restore vision without requiring genetic testing or surgical intervention.
  • Nanoscope has initiated a rolling BLA submission following positive Phase 2b/3 RESTORE trial results in retinitis pigmentosa and plans Phase 3 trials for Stargardt disease in 2025.

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

NCT04945772CompletedPhase 2
Nanoscope Therapeutics Inc.
Posted 7/13/2021

Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study

NCT06048185Active, Not Recruiting
Nanoscope Therapeutics Inc.
Posted 10/19/2023

Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

NCT05417126CompletedPhase 2
Nanoscope Therapeutics Inc.
Posted 7/5/2022

KALA BIO Names Todd Bazemore as Permanent CEO Ahead of Pivotal Phase 2b Trial Readout

Orphan DrugRare Disease
  • KALA BIO has appointed Todd Bazemore as permanent President and CEO, transitioning from his interim role since February 2025.
  • The company is approaching a critical Phase 2b CHASE trial readout for KPI-012 in persistent corneal epithelial defect, a rare orphan disease with no FDA-approved treatments.
  • Bazemore brings over 30 years of biopharmaceutical experience and will lead KALA's potential transition from clinical-stage to commercial operations.
  • KPI-012 utilizes KALA's proprietary mesenchymal stem cell secretome platform and has received Orphan Drug and Fast Track designations from the FDA.

Encoded Therapeutics Receives FDA RMAT Designation for ETX101 Gene Therapy in Dravet Syndrome

Orphan DrugRare Disease
  • The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ETX101, Encoded Therapeutics' AAV9-based gene therapy for SCN1A+ Dravet syndrome, based on preliminary seizure data from ongoing Phase 1/2 trials.
  • ETX101 is designed to increase SCN1A expression in GABAergic inhibitory neurons and already holds Fast Track, Rare Pediatric, and Orphan Drug designations from the FDA.
  • The company's new North Carolina GMP manufacturing facility has initiated ETX101 production to support pivotal trials expected in the first half of 2026.
  • Enrollment completion in the POLARIS dose-escalation clinical studies is planned for Q4 2025, with interim clinical efficacy data presentation expected during the same timeframe.

Servier and IDEAYA Biosciences Strike $530M Partnership for Uveal Melanoma Drug Darovasertib

Orphan DrugPrecision Medicine
  • Servier and IDEAYA Biosciences announced an exclusive licensing agreement worth up to $530 million for darovasertib, a protein kinase C inhibitor targeting uveal melanoma, a rare and aggressive eye cancer.
  • IDEAYA will receive $210 million upfront plus up to $320 million in regulatory and commercial milestones, while retaining US rights and sharing development costs with Servier.
  • Darovasertib has received FDA Breakthrough Therapy and Fast Track designations and is currently being evaluated in multiple Phase 2/3 trials across neoadjuvant, adjuvant, and metastatic settings.
  • The partnership aims to accelerate global development through three Phase 3 registrational trials, with median progression-free survival data expected from year-end 2025 to Q1 2026.

TME Pharma Secures €2.35M in Funding to Advance Cancer and Eye Disease Therapies

Orphan DrugOncology
  • TME Pharma completed its August financing round, raising €500,000 to bring total cash position to €2.35 million following earlier May funding.
  • The company's lead asset NOX-A12 has received FDA Fast Track Designation for glioblastoma treatment and is advancing through Phase 1/2 trials.
  • Strategic restructuring under new CEO Diede van den Ouden aims to create cost-efficient operations while developing novel cancer and ophthalmic therapies.
  • NOX-E36, the company's second lead compound, targets ophthalmic diseases requiring anti-fibrotic treatments with improved tolerability profiles.

Olaptesed With Pembrolizumab and Nanoliposomal Irinotecan or Gemcitabine/Nab-Paclitaxel in MSS Pancreatic Cancer

NCT04901741Not Yet RecruitingPhase 2
TME Pharma AG
Posted 1/1/2026

Glioblastoma Treatment With Irradiation and Olaptesed Pegol (NOX-A12) in MGMT Unmethylated Patients

NCT04121455Active, Not RecruitingPhase 1
TME Pharma AG
Posted 9/12/2019

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